Just days after Sarepta Therapeutics’ eteplirsen became the first US Food and Drug Administration (FDA) approved treatment for Duchenne muscular dystrophy, the agency’s commissioner Dr. Robert Califf is calling for the drug study to be retracted. According to Califf, the study is “misleading” and should be removed from the journal, Annals of Neurology, where it was published.
Duchenne muscular dystrophy is a rare genetic disease which causes steadily worsening muscle deterioration and results in early death. While patients and their families rejoiced following news that Sarepta’s drug was approved, the decision caused upset within the FDA with some officials questioning the drug’s ability to benefit patients.
The dispute in question is over the findings of a 2013 study of eteplirsen, which was funded by Sarepta. The study found that the drug – marketed as Exondys 51 – triggered production of the protein dystrophin, which is important in muscle integrity.
The study claimed that the injectable drug helped the patients generate enough dystrophin to allow them to walk. But Califf, along with others at the FDA, disagrees with the study authors’ interpretations.
“The publication, now known to be misleading, should probably be retracted by its authors,” said Califf in a memo on the dispute regarding accelerated approval of the drug. “… In view of the scientific deficiencies identified in this analysis, I believe it would be appropriate to initiate a dialogue that would lead to a formal correction or retraction (as appropriate) of the published report.”
Califf is not alone is his skepticism of the study. Dr. Luciana Borio, the FDA acting chief scientist said, “As determined by the review team, and as acknowledged by Dr. Woodcock, the article’s scientific findings — with respect to the demonstrated effect of eteplirsen on both surrogate and clinical endpoints — do not withstand proper and objective analyses of the data. It is very disappointing that the findings did not hold up to careful review.”
It’s unclear whether Califf and his associates’ call for the paper to be retracted will be upheld by the journal. But the journal’s editor, Dr. Clifford Saper, offered his opinion on the dispute.
“It takes more than a call by a politician for retraction of a paper,” said Saper. “It takes actual evidence.
“If you read the FDA report, you will quickly realize that it was the result of internal political squabbling within the FDA. At this point, because I am neither a politician nor a drug company representative, I have no opinion on the matter. I will wait for someone who is responsible to send me evidence of a problem, so that I can, if need be, investigate further.”